ArticleJIMD reports2025
Efficacy of Switching Therapy From Alglucosidase Alfa to Avalglucosidase Alfa on Respiratory Function in Participants With Late-Onset Pompe Disease: A Post Hoc Analysis From the COMET Trial.
Article in JIMD reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02782741 (A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa), which is not on this map. Cited by 3 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease
Who cites it
3 citing papers in PubMed.
- Real-Life Effectiveness After Switching to Avalglucosidase Alfa in Late-Onset Pompe Disease Patients Worsening on Alglucosidase Alfa Therapy: A French Cohort Study.European journal of neurology · 2026Article
- Article
- Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions.Frontiers in neurologyReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Pompe disease (PD) is a rare, autosomal recessive neuromuscular disorder caused by a deficiency in acid α-glucosidase. In the Phase 3 COMET trial (NCT02782741), respiratory function was examined in participants with late-onset PD randomized to receive enzyme replacement therapy with alglucosidase alfa (ALG; standard of care) or avalglucosidase alfa (AVA; intervention) in the primary analysis period (PAP). The open-label extended treatment period (ETP) provided long-term data on AVA treatment. This post hoc analysis evaluated respiratory outcomes in participants who received ALG in the PAP (Weeks 0-49) and switched to AVA in the ETP (Weeks 49-145). Participants were categorized according to improvement in upright forced vital capacity percent-predicted (FVC) at Week 49. Forty-four participants were included, of whom 20 (45.5%) had ΔFVC > 0% predicted at Week 49. Among ΔFVC > 0% predicted participants, FVC improved during the PAP (estimated slope [SE]: 4.0 (1.1) %/year,
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Registered trials
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